FDA Grants Breakthrough Therapy Designation to Mirum's Volixibat for Cholestatic Pruritus in PBC

Mirum Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has awarded Breakthrough therapy designation to their drug, volixibat, as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). This designation was granted following positive interim results from the Phase 2b VANTAGE study, which demonstrated statistically significant improvements in pruritus symptoms compared to a placebo for patients treated with volixibat. Publish Date: 10-10-2024 Source: Mirum Pharmaceuticals, Inc. Primary biliary cholangitis (PBC), once known as primary biliary cirrhosis, is a chronic ailment involving progressive damage, inflammation, and eventual destruction of the liver’s small bile ducts. Bile, a crucial digestive fluid produced by the liver, plays a vital role in processing food and expelling toxins, cholesterol, and aged red blood cells from the body. However, when these bile ducts suffer harm, bile accu...